P1292: BASILIXIMAB MAY BE THE POTENTIAL SOLUTION FOR SEVERE LIVER CHRONIC GRAFT-VERSUS-HOST DISEASE: A PROSPECTIVE PILOT STUDY
Authors: Dao-Xing Deng, Meng-Zhu Shen, Xiao-Hui Zhang, Lanping Xu, Yu Wang, Chen-Hua Yan, Yu-Hong Chen, Huan Chen, Wei Han, Feng-Rong Wang, Jing-Zhi Wang, Xiao-Jun Huang, Xiao-Dong Mo
Journal: HemaSphere (2023)

Topic: 22. Stem cell transplantation - Clinical Background: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the most important curative method for hematologic malignancies and non-malignant hematologic disorder. However, chronic graft-versus-host disease (cGVHD) is inevitable. The clinical outcomes of patients with severe liver cGVHD are poor, and the 5-year probability of overall survival (OS) is only 30% for these patients. Corticosteroid combined with calcineurin inhibitor (CNI) is the first-line treatment for severe liver cGVHD, but the response rate is only about 30%. Thus, the intensity of traditional initial immunosuppressive therapies is not strong enough for most of the patients with severe liver cGVHD. There is no standard second-line treatment. Thus far, there is no commonly accepted treatment for patients with severe liver cGVHD, and which drug used on the basis of steroid and CNI could help to further control severe liver cGVHD is still unknown. Interleukin-2 (IL-2) antagonist, which can inhibit the T cell activation effectively. We observed that the overall response rate (ORR) of basiliximab treatment based on corticosteroid and CNI was 56.1% in patients with liver acute GVHD. However, no study had identified the efficacy of basiliximab in patients with cGVHD. Aims: In this prospective, pilot study, we aimed to identify the efficacy and safety of basiliximab treatment on the basis of corticosteroid and CNI in patients with severe liver cGVHD. Methods: Patients who received allo-HSCT at Institute of Hematology, Peking University (PUIH) from January 2020 to May 2022 were eligible. Patients with severe liver cGVHD receiving basiliximab treatment were enrolled as basiliximab group. Patients with severe liver cGVHD but did not receive basiliximab treatment during the same time period were used as control. The last follow-up time was November 30, 2022. The primary endpoint was ORR, and the secondary endpoint included complete response (CR), toxicities, and OS. Mann-Whitney U test was used to compare continuous variables, while χ2 and Fisher’s exact tests were used to compare categorical variables. Kaplan-Meier method was used for survival analysis. P < 0.05 was considered statistically significant. The SPSS 27 and the R software package were used for data analysis. Results: Sixteen out of 20 patients (80.0%) achieved ORR after basiliximab treatment. The median time from liver cGVHD diagnosis to achieving ORR and from basiliximab treatment beginning to achieving ORR was 32 (range 10 to 74) days and 20.5 (range 2 to 54) days, respectively. Fourteen patients (70.0%) achieved CR. The median time from liver cGVHD diagnosis to achieving CR and from basiliximab treatment beginning to achieving CR was 63 (range 32 to 210) days and 51.5 (range 24 to 196) days, respectively. All characteristics were comparable between the basiliximab group and control group. The rate of ORR (80.0% vs. 40.0%, P = 0.045) and CR (70.0% vs. 20.0%, P = 0.019) in patients receiving basiliximab treatment was both significantly higher compared with those in the control group. In addition, the patients in basiliximab group showed a higher 2-year probability of OS (84.4%, 95% CI 68.1 to 100.0%) compared with those in the control group (40.0%, 95% CI 9.6 to 70.4%, P = 0.037). The hematological and infectious toxicities were all comparable between basiliximab group and control group. Summary/Conclusion: Basiliximab treatment administered on the basis of corticosteroid and CNI helped to effectively control the severe liver cGVHD, and basiliximab may provide a potential solution for these patients.Keywords: Allogeneic hematopoietic stem cell transplant, Liver, Chronic graft-versus-host, Bilirubin